Therapy Areas: Hereditary Disorders
Vertex to Submit Cystic Fibrosis Medicines Orkambi as Well as Symkevi to be Used in Combination with ivacaftor, to the Scottish Medicines Consortium for Appraisal
21 December 2018 - - US-based biotechnology company Vertex Pharmaceuticals (Europe) Ltd., following constructive discussions with the Scottish government, it will submit Orkambi (lumacaftor/ivacaftor) as well as Symkevi (tezacaftor/ivacaftor) to be used in combination with ivacaftor, to the Scottish Medicines Consortium for appraisal, the company said.

If accepted by the SMC for use on the NHS in Scotland, eligible patients with cystic fibrosis in Scotland could have access to these precision medicines in 2019.

While the SMC reviews the submissions, clinicians could apply for access to lumacaftor/ivacaftor as well as tezacaftor/ivacaftor in combination with ivacaftor for individual patients based on clinical need via the Peer Approved Clinical System Tier 2 process for 'individual access to medicines not yet generally available on the NHS'.

Vertex and the Scottish government have also agreed to a confidential discount which will be applied to applications to the PACS Tier 2 process.

Approximately 900 people in Scotland live with CF.

In the UK, the median age of death is 31 years.2 NHS Scotland estimates that one in 24 Scots have a CFTR mutation which, if carried by both parents, would lead to a child being born with CF.

Should the appraisal be positive, patients in Scotland will join those in other countries around the world where all those eligible have access to lumacaftor/ivacaftor, including Austria, Australia, Denmark, Germany, the Republic of Ireland, Italy, the Netherlands, Sweden and the US.

Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing cystic fibrosis transmembrane conductance regulator protein resulting from mutations in the CFTR gene.

Children must inherit two defective CFTR genes -- one from each parent -- to have CF. There are approximately 2,000 known mutations in the CFTR gene.

Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface.

The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs.

In the lungs, this leads to the build-up of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death.

In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface.

Patients with two copies of the F508del mutation are easily identified by a simple genetic test.

Lumacaftor/ivacaftor is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. Lumacaftor/ivacaftor is typically taken twice per day.

Some mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface.

Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases.

In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.

Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District.

Today, the company has research and development sites and commercial offices in the United States, Europe, Canada, Australia and Latin America.

Vertex is consistently recognized as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for nine years in a row.
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